MaxCyte, Inc.’s Post

New Guidances Say Gene Therapies Require Strong Preclinical Testing . . Strong nonclinical testing should underpin development programs for both gene editing (GE) products and chimeric antigen receptor (CAR-T) cellular products, according to two new final guidances issued by the FDA. Since both of these therapeutics involve genetic alterations of living human cells and potentially, the introduction of viral vectors, sponsors need a firm foundation of in vitro and in vivo testing showing that they are safe and exert limited off-target activity, both guidances say. CAR-T for non-oncology indications is not covered. The guidance “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products” focuses on the manufacturing and characteristics of CAR-T cells for oncology indications, including hematologic malignancies and solid tumors. Gene editing products are complex products that can include the gene editor, DNA targeting elements and a donor DNA template, according to “Human Gene Therapy Products Incorporating Human Genome Editing. #newguidance #genetherapy #preclinicaltesting #regulation #regulationupdate #regulatorycompliance

#Pfizer reported results from a phase 3 trial, #CIFFREO, evaluating an investigational recombinant adeno-associated virus #genetherapy known as fordadistrogene movaparvovec among ambulatory boys ages 4 to 7 years with #Duchenne muscular dystrophy. CIFFREO failed to hit its primary goal of improving #motorfunction when compared to placebo at one year of treatment, as assessed by a movement scale known as the #NorthStarAmbulatoryAssessment. Key secondary endpoints, such as 10-meter run/walk velocity and #timetorise, also failed to demonstrate a significant difference between fordadistrogene movaparvovec and placebo. These results come a little more than a month after Pfizer reported the death of a young boy participating in a related phase 2 trial called DAYLIGHT, which included boys with DMD ages 2 to 3 years. The boy had received the gene therapy in early 2023 and died from cardiac arrest on May 3 of this year. It will be interesting to review more detailed results from both of these studies to better understand the reasons why the trial failed and more importantly, whether the death of the young boy was related to the treatment. These learnings will be critical in improving future #clinicalresearch and #development of gene therapies for DMD. #DMD #AAVgenetherapy #musculardystrophy #adverseevent #drugdevelopment #challenges

SalioGen is in the News: Check out this feature article in GEN with Jason Cole, CEO, discussing our Gene Coding technology, ASGCT data presentations, and more.

🧬 Regeneron Pharmaceuticals and Intellia Therapeutics are taking a significant step forward in medical research. They're broadening their collaboration to create in vivo CRISPR-based gene editing therapies, with a focus on tackling neurological and muscular diseases.The potential of this partnership could reshape the future of medical treatments. Interested in the breakthroughs of gene editing?👉 Click the link below for the full story! 📲  https://lnkd.in/eHeKE2-b  #Regeneron #Intellia #CRISPR #GeneEditing #MedicalInnovation #NeurologicalDiseases #MuscularDiseases #MedicalResearch #TherapeuticAdvancements #FutureOfMedicine

With 15 patients started across its three gene therapies, bluebird bio claims a nearly 140% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year. The Massachusetts-based biotech revealed that it has so far initiated 15 patients on its gene therapies, one of whom started the recently approved sickle cell disease treatment Lyfgenia just this month. The remaining 14 patients are receiving either Zynteglo, for pediatric beta-thalassemia, or Skysona, which is intended for cerebral adrenoleukodystrophy in boys. William Blair analyst Sami Corwin, Ph.D. anticipates that Lyfgenia will face stiff competition from CRISPR Therapeutics and Vertex Pharmaceuticals’ Casgevy, which the analyst predicts will be a “headwind” for bluebird. One key advantage for Casgevy is its price. Bluebird gave Lyfgenia a price tag of $3.1 million, nearly $1 million more than Casgevy’s $2.2 million. #biotech #biopharma #sicklecelldisease #genetherapy

Casgevy gets the green light for sickle cell disease and beta thalassemia using CRISPR/Cas9 technology. A groundbreaking one-time treatment, it's a game-changer for blood disorders. However, don't expect a flood of approvals soon – most CRISPR treatments are in early stages. According to GlobalData, only 12% are in Phase II, one in Phase III. Analysts suggest another CRISPR approval might not be on the horizon soon. Exciting times for gene therapy, but patience is key! #Casgevy #GeneTherapy #OBI4You https://zurl.co/PKWc

"Gene therapy has given me the ability to take full control of my life... in a world where the deck was stacked against me, gene therapy has been a winning hand" - Jimi Olaghere, one of the first patients in Vertex’s exa-cel trial. An interesting article published yesterday on this week's FDA expert advisory panel for exa-cel, covering discussions held by the panel on approaches by Vertex to quantify off-target risk, suggested further safety studies, and feedback from a handful of people who have received the therapy so far. The CRISPR 2.0 Congress (Nov 28-30, Boston) will be tackling head-on challenges with safety studies and off-target assessing/predicting/monitoring. Attendees will also have the chance to leverage 17 new data readouts and 16 new tech pieces from the likes of CRISPR Therapeutics, Editas Medicine, Caribou Biosciences, Beam Therapeutics, Prime Medicine, Inc., etc. 📣 You can save $500 if you register before the EOD Friday, Nov 3. Check out pricing options here: https://ter.li/ad72fn #CRISPR #GeneEditing #Conference https://lnkd.in/eHE-WmRk

Q&A: Decentralising cryopreservation to improve cell and gene therapy trial logistics As the cell and gene therapy (CGT) pipeline continues to grow, clinical trial sites need to use better technology and increase their capacity to run more complex trials. Some sites do not have the funds to invest in services such as cryopreservation that are needed for a number of CGT trials, resulting in sponsors and biotechs avoiding these sites. OrganaBio is a company attempting to change that. Chief business officer Dr. Priya Baraniak, Ph.D. says the company has plans to launch services in more areas of the US to decentralise this process such that more sites can operate CGT trials without needing the capabilities in house, before going global. - https://lnkd.in/e7sCkTmv #cellandgene #cellandgenetherapy #celltherapy #genetherapy #clinicallogistics #pharmasupplychain #cryopreservation

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Edgewood's $20M Series A; Nanoscope to pursue FDA approval for eye gene therapy later this year: Plus, news about bluebird bio, Q32 Bio and Aquestive Therapeutics: Edgewood Oncology’s $20M Series A: The Brookline, MA-based biotech plans to use the money to support Phase 2a studies in acute myeloid leukemia and targeted breast cancer. It’s financed by Alta Partners. — Kyle LaHucik Nanoscope to seek FDA approval for eye gene therapy: The Dallas-based biotech said its mutation-agnostic gene therapy MCO-010 passed a Phase 2b study in retinitis pigmentosa, significantly improving vision measured on an eye chart at one year at both the high and low doses compared to a sham control. It expects to submit an application for MCO-010 to the FDA in the second half of this year. — Lei Lei Wu Bluebird bio projects 85 to 105 patient starts for its gene therapies in 2024: So far, the company has reported seven patient starts for beta thalassemia treatment Zynteglo and two for cerebral adrenoleukodystrophy therapy Skysona. Bluebird said that the first patient start for its sickle cell disease therapy is “imminent.” In its Tuesday earnings announcement, the company also shared that it signed an outcomes-based agreement with Michigan Medicaid, marking its first such agreement with a government payer. — Lei Lei Wu Q32 Bio begins trading: The mid-stage immune biotech completed its reverse merger with Homology Medicines and announced a $42 million private placement. Its shares $QTTB were down about 4% on Tuesday morning, its first day of trading on the Nasdaq. — Kyle LaHucik Aquestive Therapeutics closes $75M offering: The New Jersey biotech, which is developing an oral film to deliver medications, raised the money via a sale of 16.6 million shares at $4.50 apiece. — Kyle LaHucik #lucidquest #genetherapy #celltherapy

2024 is brimming with potential for cell and gene therapy 🧬 CGTLive has summarized the latest developments in CGT so far this year, including: 1️⃣ The FDA has approved Kite Pharma’s manufacturing change for Yescarta®, which reduces turnaround time – from leukapheresis to product release – from 16 to 14 days 2️⃣ Janssen and Legend Biotech’s Carvykti® therapy has moved forward for FDA review, potentially expanding its use in multiple myeloma 3️⃣ Guidelines for the development of CAR-T and genome editing products have been finalized by the FDA 4️⃣ Astellas and Mass General Brigham have embarked on a 5-year strategic collaboration in oncology, rare disease and cell and gene therapy to advance novel therapies toward commercialization It's encouraging that we are seeing developments across the CGT development, including a focus on manufacturing and scalability. We look forward to seeing further progress and impact through the rest of 2024. Read the full report ⬇️ https://lnkd.in/eBdrEKD9 #CellTherapy #GeneTherapy #AroundTheHelix #ManufacturingBrighterFutures #Biotechnology